Prospects for gene therapy using haemopoietic stem cells

Gene therapy has thus far promised much and delivered little. Much of this has been due to deficiencies in the reagents and methodologies employed in early clinical trials. Recent technological advances in vectors and haemopoietic stem cell manipulation, coupled with improved pre-clinical assays of...

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Veröffentlicht in:Best practice & research. Clinical haematology 2001-12, Vol.14 (4), p.823-834
Hauptverfasser: Fairbairn, Leslie J, Ewing, Joanne C
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creator Fairbairn, Leslie J
Ewing, Joanne C
description Gene therapy has thus far promised much and delivered little. Much of this has been due to deficiencies in the reagents and methodologies employed in early clinical trials. Recent technological advances in vectors and haemopoietic stem cell manipulation, coupled with improved pre-clinical assays of gene transfer and expression in re-populating stem cells give cause for greater optimism. Here we review these advances and indicate areas requiring further development before clinical gene therapy in the haemopoietic system becomes a widely applicable treatment modality.
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subjects Animals
clinical trial
cytokine
gene therapy
Genetic Therapy - methods
Genetic Vectors - genetics
Genetic Vectors - therapeutic use
Hematopoietic Stem Cells - metabolism
Humans
pseudotyping
retroviral vector
Transduction, Genetic - methods
Transduction, Genetic - trends
title Prospects for gene therapy using haemopoietic stem cells
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